Many who have been working in this field even for a few years know that regenerative medicine has been literally a staple of daily practice. Now things are moving from the sole on up. A terrific article by Kristen Gerencher of Marketwatch outlines how.
SAN FRANCISCO (MarketWatch) — Two years ago, doctors closed a longstanding hole in Susan Dennington’s right foot using a skin graft manufactured from infant boys’ donated foreskin.
Dennington, 52, suffers from neuropathy so she couldn’t feel the pain from her diabetic foot ulcer, but the gaping wound made it hard for her to function. She spent much time and money keeping it clean and bandaged to protect it from infection. Several infections happened anyway during the 10 years her ulcer persisted, and there were times she thought the ulcer was finally going away only to have it return again.
Her doctor, Quyen Ha of Ada, Okla., said at first Dennington was skeptical about a wound-care product called Apligraf but was determined not to face amputation. “My job as a wound doctor is to save as many legs as I can,” he said.”It was completely frustrating,” she said. “My wound was on my mind every day.”
So Dennington had several outpatient procedures to apply Apligraf, followed by six weeks of recovery. While some patients with similar medical problems have to come back for more procedures because of their underlying disease, she has not returned for more treatments, said Ha, who doesn’t receive any fees from the product’s maker, Organogenesis of Canton, Mass.
“Since I’ve had the Apligraf, there’s been no treatment and it’s never come back,” Dennington said of her foot ulcer. “My wound is closed and I haven’t worn a bandage in two years. That feels like freedom.”
Dennington is the beneficiary of a quiet movement focused on shifting medical care to living-cell-based therapies that stimulate the body’s tissue to regenerate and heal itself, as opposed to more traditional approaches focused on managing symptoms. Regenerative medicine, as it’s known, encompasses a variety of scientific disciplines and includes stem-cell technologies, medical devices, biotechnology and even pharmaceutical companies.
Hoping to cure wide range of diseases
Some forms of it, such as orthopedic bone grafts and skin grafts for burn patients, have been around for years. Apligraf itself has been on the market for more than a decade. But interest in regenerative medicine is picking up as many ambitious new therapies aim to overcome some of the biggest causes of illness, disability and death, including heart failure, diabetes, Parkinson’s disease, spinal-cord injury and macular degeneration that can lead to blindness.
Some traditional drugmakers want in as well. In January, pharmaceutical giant Eli Lilly (LLY 34.86, +0.09, +0.26%) and the Juvenile Diabetes Research Foundation announced they’re teaming up to fund research aimed at helping patients with type 1 diabetes regenerate insulin-producing cells destroyed by the disease.A few examples: Advanced BioHealing makes a similar product for diabetic foot ulcers called Dermagraft. Last month, it filed for a $200 million initial public offering. Another company, Tengion(TNGN 2.64, -0.13, -4.69%), is developing methods to repair failing organs such as bladders with a combination of biocompatible materials and the patient’s own cells.
Investors take note
The market capitalization of 22 publicly-traded companies in the cellular therapy business hit $2.5 billion this year, up 40% from last year, said Robin Young, chief executive of data firm Pearl Diver and organizer of the annual New York Stem Cell Summit. He projects U.S. sales will grow to about $1 billion by 2015.
Cell-based approaches involve changing the course of diseases, said Morrie Ruffin, managing director of the Alliance for Regenerative Medicine, a trade group of 80 companies based in Bethesda, Md., that launched in 2009.
“One of the ways we’re going to have to address the long-term cost increase we face in health care is by focusing on innovation and getting out of this cycle of looking at very small incremental gains in treating diseases to finding transformative solutions,” Ruffin said. “That’s why regenerative medicine is so exciting. It takes us in that direction.”
The industry wants Congress to reintroduce the Regenerative Medicine Promotion Act of 2010, which would provide federal funds for research and development and streamline the approval process for products deemed safe and effective.
Meanwhile, Wall Street wants to see signs of clinical viability, said Mark Monane, managing director of equity research at Needham & Co. in New York.
“Investors are not giving these companies the benefit of the doubt,” he said. “They trade at a discount relative to other like-stage companies. For the most part, the jury is still out on whether this can be a product in the real world or if they are doing a science experiment.”
Even so, investors appear increasingly interested. In January, the Alliance for Regenerative Medicine held its first annual “state of the industry” briefing during the Biotech Showcase and the J.P. Morgan Healthcare Conference in San Francisco. The turnout of attendees forced administrators to shift the event to a bigger room as executives gave company overviews and took questions.
Tapping embryonic stem cells
Earlier this year, the U.S. Food and Drug Administration cleared Advanced Cell Technology (ACT) (ACTC 0.16, -0.0022, -1.32%) of Marlborough Mass., to pursue a multicenter clinical trial to treat dry age-related macular degeneration, which affects 10 million to 15 million Americans. The treatment involves inducing a cell line grown from human embryonic stem cells to become retinal pigment epithelial (RPE) cells, which sit at the base of the retina on top of what’s called the Bruch’s membrane.
“It’s the death of these RPE cells that leads to a variety of retinal disease, the biggest of which is dry age-related macular degeneration,” ACT Chief Executive Gary Rabin said.
In the upcoming trials, eye surgeons will inject the new cells into patients’ diseased eyes with the aim of having the cells graft and reverse the damage in an area of the body known for being insulated from typical immune responses that would reject the new cells, he said.
Embryonic stem cells are much more malleable than adult stem cells, Rabin said, and he expects ACT’s method to gain acceptance no matter how the politics of embryonic stem cells evolves.
In February, the company received a patent on an extraction technique called “single blastomere” that doesn’t harm the two-day-old embryos. Advanced Cell Technology acquires them from in vitro fertilization clinics with patients’ consent, uses the embryos to start cell lines and then returns them to the clinic for their original purpose — for making babies or storing for the future.
“The extraction method we use is used in the birth of thousands of babies each year using PGD [pre-implantation genetic diagnosis],” Rabin said. “We’re not changing the fate of the embryo we borrowed.”
Another company, Kinetic Concepts Inc. (KCI 51.55, +0.13, +0.25%) acquired a regenerative-medicine outfit called LifeCell in 2008. One of its flagship products, Strattice Reconstructive Tissue Matrix, used mostly for breast and abdominal wall reconstruction, is changing how surgeons practice, said Dr. Ron Silverman, a plastic surgeon and chief medical officer for Kinetic Concepts in San Antonio.
“LifeCell’s approach has been to provide a natural matrix, like scaffold, and allow your own cells go into the scaffold,” he said. “Within a day there are already cells within the product. When you have your own cells, there’s no rejection.”
Strattice provides an alternative to the synthetic mesh often used in hernia surgery, which can cause suffering and complications if infection develops, he said. With plastic, a second surgery would be required to remove the mesh whereas regenerative tissue doesn’t require invasive correction, he said. “We see this as tremendously economically advantageous.”
It’s an example of how regenerative medicine facilitates the body’s ability to grow new cells, Silverman said. “If you don’t have regeneration, then the only thing the body knows how to do is heal things by scarring. Regeneration allows not just scarring but replacement of natural tissue.”
For companies struggling to bring products to market, collaboration will be key, said Monane of Needham & Co.
“There’s so much opportunity to win,” he said. “They’re all at a stage where they’re learning from each other.”
Kristen Gerencher is a reporter for MarketWatch in San Francisco.